Catherine L’Estrange, 11, from London has become the first UK patient, and second in the world, to have a new gene therapy for a rare condition she was born with. Catherine was diagnosed bardet biedl syndrome (BBS) when she was just a baby. It is caused by mutations to one of 20 different genes and impacts around one in 100,000 births in the UK, leaving patients blind by their late teens or early 20s. It can also cause kidney problems, learning difficulties and obesity, and patients sometimes have extra fingers or toes. Surgeons performed keyhole surgery on her eye before…
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